Summary

Multiple research groups have linked brain disease such as amyotrophic lateral sclerosis (Lou Gehrig's disease), Huntington, and Alzheimer's to defects in the cellular transport system that carries proteins back and forth across the nuclear membrane. Somehow, the resulting abnormal pileups of proteins become rogue neuronal killers in those ailments. The findings promise to transform understanding of these diseases. They are also inspiring therapeutic efforts to address the cause of general age-related neurodegeneration—a goal that has largely eluded drug developers. Several biotech companies have jumped on that idea, exploring it in animal models and planning the first human trials of drugs that target intracellular transport this year.

↵*Elie Dolgin is a science journalist based in Somerville, Massachusetts.